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VX -770 (104) in People with CF age 12 and older with 2 copies of the F508del-cftr mutation

Study Type:

Interventional

Intervention Category:

CFTR Modulation

Study Sponsor:

Vertex

Study Phase:

3

Recruitment Status:

Trial Completed

Study Drug(s):

Kalydeco

Number of Participants Being Recruited:

40

Single / Multi-Center:

Multi-Center

STUDY BACKGROUND INFORMATION:

This is a 2-part Phase 2 study assessing VX 770 in people ages 12 yrs and older with two copies of the F508del-cftr mutation. In Part A subjects take VX-770 or placebo by mouth twice daily (morning and evening) for 16 weeks. Subjects who complete Part A and meet certain response criteria will be invited to enroll in Part B. Part B is an open-label extension and eligible subjects will receive VX-770 twice daily for up to 96 weeks.

ELIGIBILITY

Age:

>= 12 Years

FEV1:

>= 40 Percent Predicted

P. aeruginosa status:

Not applicable

B. cepacia status:

Not applicable

Other Primary Eligibility Requirements:

Note: Detailed eligibility criteria information may be available on clinicaltrials.gov. If a specific
trial listing for this trial is available, a link to the specific clinicaltrials.gov listing will be present in the "More Information" section below.

The changes in FEV1 or sweat chloride in Part A were not sustained with ivacaftor treatment from Week 16 to Week 40.

Safety:

Forty-two (37.5%) ivacaftor subjects and 6 (21.4%) placebo subjects from the VX-770-104 (Discover) trial (Part A) qualified for Part B, based on pre-specified eligibility criteria including either =10% change relative to baseline in FEV1 % predicted at any time point through Week 16, or a sweat chloride concentration reduction from baseline =15 mmol/L at both the Day 15 and Week 8 visits. Thirty-three subjects entered the open label follow-on: 28 of these subjects met the FEV1 criterion and 5 met the sweat chloride criterion. Of 6 placebo subjects eligible for Part B, 5 subjects enrolled; all on the basis of FEV1 changes. No new safety signals were identified.